Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I



Status:Active, not recruiting
Conditions:Metabolic, Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:18 - Any
Updated:1/25/2017
Start Date:July 2015
End Date:August 2017

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A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Adult Patients With Mucopolysaccharidosis I (MPS I, Hurler Syndrome)

AGT-181 is a fusion protein containing alpha-L-Iduronidase that is intended to deliver the
enzyme peripherally and to the brain, when administered intravenously. This study is a
safety and dose ranging study to obtain safety and exposure data, as well as information on
the biological activity of the investigational drug.


Inclusion Criteria:

- Male age 18 years or older

- Diagnosis of MPS I (documented fibroblast or leukocyte IDUA enzyme activity level of
less than 10% of the lower limit of the normal range of the measuring laboratory)

- Voluntary written consent by patient or legally responsible representative

- All women of childbearing potential and sexually mature males must be advised to use
a medically accepted method of contraception throughout the study.

- Negative pregnancy test (females)

- Must not have received ERT for at least 6 weeks prior to AGT-181 treatment

- Must have elevated urinary GAGs if no ERT has been received in the prior 3 months

Exclusion Criteria:

- Refusal to complete baseline evaluations.

- Any medical condition or other circumstances that may significantly interfere with
study compliance

- Receipt of an investigational drug within the prior 90 days

- History of diabetes mellitus or hypoglycemia

- Clinically significant spinal cord compression, evidence of cervical instability.

- Known hypersensitivity to alpha-L-iduronidase or any of the components of AGT-181.

- Known to be nonresponsive to standard ERT treatment.

- Previously successful (engrafted) hematopoietic stem cell transplantation that
resulted in normalization of urinary GAGs.

- Contraindication for lumbar puncture
We found this trial at
6
sites
1201 W La Veta Ave
Orange, California 92868
(714) 997-3000
Principal Investigator: Raymond Wang, MD
Phone: 714-509-3008
Children's Hospital of Orange County For more than 45 years, CHOC Children’s has been steadfastly...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Principal Investigator: Jerry Vockley, MD, PhD
Phone: 412-692-6065
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Barbara K Burton, MD
Phone: 312-227-6129
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Chicago, IL
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Decatur, Georgia 30033
Principal Investigator: William Wilcox, MD
Phone: 404-778-8421
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Decatur, GA
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Oakland, California 94609
Principal Investigator: Paul Harmatz, MD
Phone: 510-428-3885
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Oakland, CA
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Salt Lake City, Utah 84132
Principal Investigator: David H Viskochil, MD
Phone: 801-587-3605
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Salt Lake City, UT
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